Empowering Patients in Healthcare

“Patients are at the heart of all we do!” This is a sentence we hear over and again from industry, and yet patient engagement is often reported as a challenge by both patient groups and industry alike.  Some patient organisations feel frustrated by a lack of early engagement and are overburdened and underprepared when supporting Health Technology Appraisals¹. How can this process be improved, recognising the true value of the patient voice whilst respecting the impact of providing it?

There is no doubt that patients should be at the heart of every drug development plan.  The US Food and Drug Administration (FDA)², The European Medicines Agency (EMA)³ and the Medicines and Healthcare products Regulatory agency (MHRA)⁴ have all started to incorporate the patient voice in areas relating to clinical trial design and patient outcomes.  In rare diseases, the role of the patient is perhaps even more critical in the drug development process.  The lack of knowledge in rare diseases, specifically: clinical expertise; natural history of the disease; prevalence and incidence data; variable symptoms and multiple organ involvement can all make it challenging to develop new treatments^5,6.  However, these factors are essential in drug development to ensure that clinical development considers the most appropriate end points, reflecting the patient experience to regulatory bodies and payers.

Patient communities are becoming more involved in the process and play a key role by supporting natural history studies and quality of life measurement, that can be used to highlight any benefits of a new treatment.  Beyond the benefits to the drug development process, involvement in these types of studies can also provide opportunities for the patient community to network between one another and with the clinical experts in the field, sharing their knowledge and forming a support system.

So, let us consider some of the current challenges surrounding when and how to engage with patients?

Often patient groups are borne from the lack of existing knowledge and information available to them at the point of their diagnosis with a rare disease.  The NCBRS worldwide foundation are one such patient organisation—developed from the drive of two parents, desperate to learn more about their son’s condition and find other parents who shared their experiences.  They are now an established global patient organisation who support a patient registry, an adult research study and several other innovative research projects.  Through engaging with families near and far, they have established a thriving support network for the whole community and held their first family conference in May of this year.  However, with only 270 cases of Nicolaides-Baraitser Syndrome (NCBRS) worldwide, funding these projects has been a challenge.

The increased knowledge and understanding of a disease created by patient advocacy groups like NCBRS worldwide foundation is not only invaluable to their community but will be hugely valuable to any prospective drug development programme for the condition.  Therefore, it makes sense that supporting and funding these sorts of efforts should be a priority for researchers interested in advancing treatments in rare diseases.   For many small and newly established patient groups, starting the process of engagement and reaching out to researchers and companies may be overwhelming. 

Identifying funding sources, completing applications and working within strictly regulated funding environments requires insights, knowledge and a huge amount of time to support completion.  Time which many families have little of, as they meet the care needs, hospital visits and day-to-day support needs of their loved one. And yet, they continue to excel and establish groundbreaking projects as they are driven by the need to improve the lived experience for their communities.

We must therefore ponder, could the systems be made easier for people in the rare disease community who have the knowledge and insights to make the difference but who rarely have the time, resources or funding to drive them forward?

Currently patient organisations are heavily reliant on funding from pharmaceutical companies to resource their organisations and projects.  This has been a challenge in the current fiscal climate.  I have had several conversations with leaders of small and large patient groups working in rare, who have reported that the past 12-18 months have been an extremely challenging time for them.  Added to the fiscal climate are some of the wider challenges that have existed for some time. 

Public perception of engagement between pharmaceutical companies and patient groups is driven by misleading articles in the media.  Headlines like “Revealed: drug firms funding UK patient groups that lobby for NHS approval of medicines” published in the Gaurdian⁷, give the impression of a corrupt and unethical process in play.  These types of headlines have undoubtedly created a nervousness for both patient groups and pharmaceutical companies alike, making them perhaps more reluctant to engage.

In stark contrast to these news headlines, patient engagement with pharmaceutical companies is tightly regulated. For example, within the UK, these types of engagement are regulated by the ABPI code of conduct, which lays out clear guidance on how companies can engage and contract with patient groups⁸.  Companies have their own strict guidance on patient engagement, which often go much further and are stricter than guidelines laid down by organisations like the ABPI. 

When patient groups do enter contracts with pharmaceutical companies, they may find themselves subject to the ABPI code of conduct and the companies own legal terms of engagement, creating a legal minefield for both parties.  Without pro-bono legal support, the reality is that many small patient organisations may find the process of contracting with companies on specific projects too overwhelming.

We must create a system where patient engagement in drug development is not only encouraged but is seen as essential.  Perhaps we need to look at some of the things that are creating barriers.  For example, what drives the media headlines that create this understandable anxiety about engagement?  Perhaps we need to educate the public and the media about the critical role of patient engagement in the drug development process.  This may help to remove these sensationalist headlines from our mainstream media, replacing them of stories about patient engagement leading to innovation and drug discovery.  Hopefully the Rare Disease Research Network,which will launch in November 2024,  will provide an opportunity for patient organisations to put forward their innovative ideas for research in a forum where academics and commercial researchers can support them and take their ideas forward. 

Perhaps the thing that could have the potential to make the biggest difference for patient groups of all sizes, would be the creation of a centrally held fund open to all patient groups regardless of their size, their stage of development or their ambitions.  Having access to this type of funding system would remove the anxiety surrounding engagement between patient groups and pharma as the pot would be held by an independent body.  There would be no suggestion of companies funding groups who are involved in their disease area of interest, the burden on patient groups could be reduced through a simplified and standardised application process and open to all patient groups whether there is a medicine in development or not. 

This type of pot could surely create opportunities for innovation to thrive, allow patients to be front and centre of providing the data that matters most in the drug development process and remove some of the challenges of patient engagement.

Of course, there are many other challenges surrounding patient engagement and it would be naïve of me to suggest that these aspects alone could fix the problems. However, creating an environment where including the patient voice in drug development is simple, clear and without anxiety would be a first step towards true patient centricity.

1. realise-advocacy-pilot-report-supporting-patients-in-drug-development-and-hta.pdf (secure.website)
2. FDA (2024) CDER Patient -Focused Drug Development Available at: CDER Patient-Focused Drug Development | FDA [Accessed 15 July 2024]
3. https://www.ema.europa.eu/en/documents/other/executive-summary-patient-experience-data-eu-medicines-development-and-regulatory-decision-making-workshop_en.pdf
4. MHRA pilots patient involvement in new applications – GOV.UK (www.gov.uk)
5. UK Bioindustry Association, 2014. Very rare diseases, complex issues: evaluating orphan and ultra-orphan drugs. Available at: https://www.bioindustry.org/resource/very-rare-diseases-complex-issues-evaluating-orphan-and-ultra-orphan-drugs.html [Accessed 2 September 2024]
6. Fermaglich,L. & Miller, K., 2023. A comprehensive study of the rare diseases and conditions targeted by orphan drug designations and approval over the forty years of the Orphan Drug Act. Orphanet Journal of Rare Diseases. 18:163. Available at: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10290406/#:~:text=Researchers%20have%20estimated%20that%20there%20may%20be%207000%E2%80%9310%2C000,shown%20promise%20in%20their%20treatment%2C%20diagnosis%20or%20prevention [Accessed 2 September 2024]
7. Revealed: drug firms funding UK patient groups that lobby for NHS approval of medicines | Drugs | The Guardian
8. ABPI 2021 Code of Practice
9. Home – Rare Disease Research Network (rd-rn.org)

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